Sickle cell
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News
HSCT | Briquilimab Granted Orphan Drug Designation By The European Commission
Previously, the U.S. Food and Drug Administration (FDA) granted orphan drug designation to briquilimab in HCT, as well as rare pediatric disease designation for the treatment of severe combined immunodeficiency…
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TIF Releases New Scientific Bulletin To Highlight Research On Haemoglobin Disorders
A key objective of the Thalassaemia International Federation is to inform and update regularly the global haemoglobinopathies family, both patients and physicians, on the progress and new developments that arise…
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News
COMING UP: New TIF Webinar On ”COVID-19 Vaccinations & Patients With Haemoglobin Disorders”
TIF is delighted to inform you that a Special Webinar on ‘’COVID-19 Vaccinations & Patients with Haemoglobin Disorders’’ will take place on Wednesday, 31 March 2021, at 14:30-16:00 EEST/12:30-14:00 GMT.…
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Clinical Trial Cleared for GPH101: The First Potentially Curative Sickle Cell Disease Therapy
The U.S. Food & Drug Administration (FDA) has cleared an investigational new drug (IND) application for the experimental gene editing therapy GPH101 by Graphite Bio to initiate a Phase 1/2…
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EU Approves Adakveo to Treat Vaso Occlusive Crises in Sickle Cell Disease Patients
The European Commission has approved Novartis’ Adakveo (crizanlizumab) as a preventive treatment for recurrent Vaso-Occlusive Crises (VOCs) in patients, 16 and older, with sickle cell disease (SCD). This approval follows a recommendation for conditional approval issued in July…
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Gene Therapy LentiGlobin Granted Priority Medicines Designation for Sickle Cell Disease in the EU
Bluebird Bio’s investigational gene therapy LentiGlobin has been granted priority medicines (PRIME) designation by the European Medicines Agency (EMA) for the treatment of sickle cell disease (SCD). Given to candidate therapies with promising clinical data for diseases of high…
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Coronavirus Disease among Persons with Sickle Cell Disease
Individuals with sickle cell disease (SCD) are a particularly vulnerable group of patients, with a higher risk of severe complications due to COVID-19 infection than the general public. A recent…
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GBT Announces Plans to Seek Regulatory Approval for Oxbryta® (Voxelotor) to Treat Sickle Cell Patients in Europe
Global Blood Therapeutics (GBT) plans to submit a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for Oxbryta (Voxelotor) be approved for treating hemolytic anemia in patients with sickle cell disease…
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COVID-19 & Haemoglobin Disorders: The Impact of Coronavirus on Sickle Cell Disease Patients
An Expert Panel of Six Medical Professionals from various countries, such as India, the UK, Italy and Saudi Arabia, shared their experiences with TIF on the impact of the COVID-19…
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